Could a Two-Drug cocktail tame ALS? early trial hints at hope
NCT ID NCT06307301
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tested whether combining two drugs—abatacept and interleukin-2—is safe for people with ALS. The goal was to boost the body's regulatory T cells, which may help slow disease progression. Only 5 participants were enrolled, and the study focused on safety and immune system changes, not on curing the disease.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- abatacept (Orencia) and interleukin-2 (aldesleukin)
- What this could lead to
- If safe and effective, this combination could help slow ALS progression by boosting the body's own immune regulation.
- What could go wrong
- This is a very early, small phase 1 trial with only 5 participants, so results may not apply to all ALS patients. Side effects from the drugs are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
-
5 people
The number who actually took part.
- Started
-
Oct 2021
- Finished
-
Dec 2023
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 years and older
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: Patients will be eligible for initial enrollment on this study if they meet the following criteria at the time of screening: 1. Provided informed consent and authorized use of protected health information (PHI) in accordance with national and local patient privacy regulations. 2. ALS meeting El Escorial criteria for possible, probable, lab-supported probable, or definite ALS. 3. At least 18 years old. 4. Total bilirubin less than or equal to 1.5 mg/dL 5. Alanine aminotransferase level (ALT) less than or equal to five times normal, albumin greater than or equal to 3.0 gm/dL 6. Serum creatinine less than 1.5 mg/dL 7. Capable of complying with all study procedures, including the study drug delivery procedure, in the Investigator's opinion. 8. A family member or caretaker who is expected to be consistently available to administer both study drugs of abatacept and IL-2 if the participant is unable to do so. 9. On a stable regimen of riluzole for at least 30 days at the time of screening. If not on riluzole at the time of study entry, willing to refrain from initiation of the agent for the duration of the trial. 10. Patients on edaravone willing to refrain from taking edaravone on the same day as they will receive the abatacept injection for the duration of the trial. If not on edaravone at the time of study entry, willing to refrain from initiation of the agent for the duration of the trial. 11. Forced vital capacity (FVC) ≥50% of predicted capacity for age, height, and sex at screening, or receiving treatment with noninvasive ventilation if FVC \< 50% of predicted for age, height, and sex at screening. Exclusion Criteria: Patients will be ineligible to participate if any of the following are true at the time of screening: 1. Serious, active bacterial, fungal, or viral infection, active or latent tuberculosis. 2. Tracheostomy. 3. Severe cardiac dysfunction defined as left ventricular ejection fraction \<40% if an echocardiogram is medically indicated to clarify ongoing symptoms or EKG findings.; a history of non-controlled cardiac arrhythmias; history of cardiac tamponade; Unstable angina or MI in the last 3 months. 4. Hypersensitivity or allergy to IL-2 or abatacept. 5. History of bowel ischemia/perforation, or GI bleeding requiring surgery. 6. History of resistant seizures, history of coma or toxic psychosis lasting \>48 hours. 7. Platelets \<100,000/mm3; hematocrit \<30%. 8. History of cancer in the past 5 years (except cutaneous Basal cell carcinoma or squamous cell carcinoma). 9. Hx of immunomodulation therapy including IL-2 or abatacept administration in the past 90 days. 10. Treatment with another investigational drug, biological agent, or device within 30 days or 5 half-lives of screening, whichever is longer. 11. If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or unwilling to use effective contraception for the duration of the trial and for 90 days after treatment. 12. If male of reproductive capacity, unwilling to use effective contraception for the duration of the trial and for 90 days after treatment
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Amyotrophic lateral sclerosis are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Houston Methodist Research Institute
Houston, Texas, 77030, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- A randomized, placebo-controlled, multicenter, clinical trial of colchicine in amyotrophic lateral sclerosis
- Interplay between gut microbiota and adaptive immunity in amyotrophic lateral sclerosis: a clinical trial
- A multicenter, open-label extension (OLE) study to evaluate the safety, pharmacodynamics, and clinical effects of WVE-004 in patients with C9orf72-associated amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD)
- Contrasting chronic inflammation and neurodegeneration using the human amniotic mesenchymal cell secretome as an innovative therapeutic strategy
- Can a brain implant let paralyzed people control computers with their minds?
- AI hunts for early clues to Parkinson's and Alzheimer's progression